The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use. In AML, the decision was based on data from the phase III RATIFY trial, in which patients with newly diagnosed AML who were treated with midostaurin had a 23 percent lower risk of death (P value not provided) than patients who received a placebo. Midostaurin treatment also extended patients’ median overall survival (74.7 months vs. 25.6 months; hazard ratio = 0.77 [95% CI 0.63-0.95]; p=0.0078). The most common adverse events (AEs; occurring in ≥30% of patients) associated with midostaurin included febrile neutropenia, nausea, exfoliative dermatitis, vomiting, headache, petechiae, and pyrexia.

The recommendation for advanced systemic mastocytosis was based on two single-arm, open-label studies in which midostaurin-treated patients had an overall response rate of 59.6 percent (95% CI 48.6-69.8). The most common AEs associated with midostaurin in the mastocytosis studies were nausea, vomiting, diarrhea, peripheral edema, and fatigue.